IRA Effect: Alnylam Acting ‘Rationally’ In Halting Second Orphan Indication For Amvuttra – Analysts

Alnylam decision has been seized by opponents of Medicare price negotiation as evidence the nascent program will undermine innovation, particularly for small molecule drugs.

Price Controls In The Law Driving Shifts In Pipeline Plans • Source: Shutterstock

Rare disease treatment developer Alnylam Pharmaceuticals Inc. has scrapped plans to begin a Phase III study on use of its second-generation RNAi product Amvuttra (vutrisiran) in a second orphan drug indication because having more than one orphan indication could make the drug subject to Medicare price negotiation under the Inflation Reduction Act.

The study would have investigated use of vutrisiran in Stargardt disease, a rare genetic eye disorder that can cause vision...

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